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Niemann-Pick type C disease
Biotech
Cyclo Therapeutics merges with longtime backer Rafael Holdings
Cyclo’s lead asset is Trappsol Cyclo, a formulation of hydroxypropyl beta cyclodextrin being evaluated in Niemann-Pick disease type C1.
James Waldron
Aug 22, 2024 8:50am
'Clinical intuition' led FDA advisors to back Zevra's rare disease med
Aug 5, 2024 10:35am
Orphazyme meme ends with $13M sale to satisfy bankruptcy court
May 16, 2022 6:06am
Roche-partnered Scenic nabs $31M to test 3 rare disease meds
Mar 10, 2022 10:12am
Orphazyme to assess strategic options after EU advisers nix drug
Feb 24, 2022 10:45am
Pfizer, NEA-backed rare disease startup bumps up Series A to $42M
Jul 25, 2016 10:32am